Betain and haemophilia A

BetHäm2012: A monocenter, single-blind, randomized, 2-armed, placebo-controlled, cross-over study to investigate coagulation markers in patients with haemophilia A after administration of Betain

Status

Active, not recruiting

Phases

Phase 1Phase 3

Study type

Interventional

Source

EU CTR

Registry ID

EUCTR2012-001039-30-DE

Enrollment

Registered

2012-11-16

Start date

2013-03-06

Completion date

Unknown

Last updated

2025-03-10

For informational purposes only — not medical advice. Sourced from public registries and may not reflect the latest updates. Terms

Conditions

In this study patients with haemophilia A are included. MedDRA version: 18.1 Level: LLT Classification code 10018937 Term: Haemophilia A System Organ Class: 100000004850

Interventions

Trade Name: Cystadane Product Name: Betaine anhydrous Pharmaceutical Form: Powder for oral solution INN or Proposed INN: Betaine anhydrous CAS Number: 107-43-7 Other descriptive name: BETAINE Concent

Eligibility

Sex/Gender

Male

Inclusion criteria

Inclusion criteria: Male patients with moderate or mild haemophilia A at the age of 18 – 70 years without an inhibitor against FVIII Written informed consent No need for regularly prophylactic treatment with FVIII product Complaince of the patient Are the trial subjects under 18? no Number of subjects for this age range: F.1.2 Adults (18-64 years) yes F.1.2.1 Number of subjects for this age range 25 F.1.3 Elderly (>=65 years) yes F.1.3.1 Number of subjects for this age range 5

Exclusion criteria

Exclusion criteria: Patients without haemophilia A or patients with haemophilia A at the age of under 18 years or older than 70 years Known adverse reactions against Betain substrate or components of Betain or placebo Known history of cerebral oedema Legal incompetence Participating in other clinical studies Comedication which would interfere to administration of Betain

Design outcomes

Primary

Measure	Time frame
Main Objective: Primary endpoint is FVIII activity;Secondary Objective: Secondary endpoints are aPTT VWF-Ag VWF-Akt FIX:C FVIII inhibitor Methionin level CAT Absence of adverse events ;Primary end point(s): Primary endpoint is FVIII activity;Timepoint(s) of evaluation of this end point: Screening day start of IMP-intake (U1) day 1 after start of IMP-intake (U2) day 3 after start of IMP-intake (U3) day 7 after start of IMP-intake (U4) day 14 after start of IMP-intake (U5) day 1 after start wash-out (U6) day 3 after start wash-out (U7) day 7 after start wash-out (U8) day start IMP2-intake (U9) day 1 after start of IMP2-intake (U10) day 3 after start of IMP2-intake (U11) day 7 after start of IMP2-intake (U12) day 14 after start of IMP2-intake (U13) day 1 after end of IMP-intake (U14) day 3 after end of IMP-intake (U15)	—

Measure

Time frame

Main Objective: Primary endpoint is FVIII activity;Secondary Objective: Secondary endpoints are aPTT VWF-Ag VWF-Akt FIX:C FVIII inhibitor Methionin level CAT Absence of adverse events ;Primary end point(s): Primary endpoint is FVIII activity;Timepoint(s) of evaluation of this end point: Screening day start of IMP-intake (U1) day 1 after start of IMP-intake (U2) day 3 after start of IMP-intake (U3) day 7 after start of IMP-intake (U4) day 14 after start of IMP-intake (U5) day 1 after start wash-out (U6) day 3 after start wash-out (U7) day 7 after start wash-out (U8) day start IMP2-intake (U9) day 1 after start of IMP2-intake (U10) day 3 after start of IMP2-intake (U11) day 7 after start of IMP2-intake (U12) day 14 after start of IMP2-intake (U13) day 1 after end of IMP-intake (U14) day 3 after end of IMP-intake (U15)

—

Secondary

Measure	Time frame
Secondary end point(s): Secondary endpoints are aPTT VWF-Ag VWF-Akt FIX:C FVIII inhibitor Methionin level CAT Absence of adverse events;Timepoint(s) of evaluation of this end point: Screening day start of IMP-intake (U1) day 1 after start of IMP-intake (U2) day 3 after start of IMP-intake (U3) day 7 after start of IMP-intake (U4) day 14 after start of IMP-intake (U5) day 1 after start wash-out (U6) day 3 after start wash-out (U7) day 7 after start wash-out (U8) day start IMP2-intake (U9) day 1 after start of IMP2-intake (U10) day 3 after start of IMP2-intake (U11) day 7 after start of IMP2-intake (U12) day 14 after start of IMP2-intake (U13) day 1 after end of IMP-intake (U14) day 3 after end of IMP-intake (U15)	—

Measure

Time frame

Secondary end point(s): Secondary endpoints are aPTT VWF-Ag VWF-Akt FIX:C FVIII inhibitor Methionin level CAT Absence of adverse events;Timepoint(s) of evaluation of this end point: Screening day start of IMP-intake (U1) day 1 after start of IMP-intake (U2) day 3 after start of IMP-intake (U3) day 7 after start of IMP-intake (U4) day 14 after start of IMP-intake (U5) day 1 after start wash-out (U6) day 3 after start wash-out (U7) day 7 after start wash-out (U8) day start IMP2-intake (U9) day 1 after start of IMP2-intake (U10) day 3 after start of IMP2-intake (U11) day 7 after start of IMP2-intake (U12) day 14 after start of IMP2-intake (U13) day 1 after end of IMP-intake (U14) day 3 after end of IMP-intake (U15)

—

Countries

Germany

Contacts

Public ContactDr. med. W. Miesbach, Med. Klinik I

Hospital of Goethe-University

wolfgang.miesbach@kgu.de00496963017788

Outcome results

None listed

Source: EU CTR (via WHO ICTRP) · Data processed: Feb 4, 2026